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Akcea steals march on Alnylam with NICE approval

Advantage to Tegsedi over Onpattro


NICE has recommended Akcea Therapeutics’ rare disease treatment Tegsedi (inotersen), after the company improved its price offer.

The draft decision from NICE’s highly specialised technologies (HST) workstream is a major boost for Akcea, which was initially rejected in December alongside its close rival, Alnylam’s Onpattro (patisiran).

Both treatments are for hereditary transthyretin-related amyliodsis (hATTR) an ultra-rare progressive condition that causes the liver to produce abnormal transthyretin protein, and which affects just 150 or so people in the UK.

NICE has announced today that it has been able to change its mind on Tegsedi and issued a positive recommendation after Akcea (a subsidiary of Ionis) offered an improved commercial agreement.

England’s cost effectiveness watchdog says this has addressed the “uncertainties in the company’s economic model” - but Alnylam’s Onpattro is still awaiting the same green light.

It is as-yet unclear whether Alnylam will take days, weeks or months longer to reach its own agreement with NICE, but gaining NICE recommendation ahead of its rival is great news for Akcea.

This is vital for Tegsedi, which had generally been seen as the runner-up to Onpattro in terms of the data produced in their pivotal clinical trials.

While the drugs represent a major step forward in improving the outlook for patients with hATTR, they do represent a major expense for healthcare systems, as they require lifelong treatment.

Tegsedi is self-administered once weekly by subcutaneous injection and has a list price of £5,925 per 284 mg weekly dose, though this will have been significantly reduced in the confidential deal struck between the company and the NHS.

By contrast, Onpattro is administered once every three weeks intravenously, and has the higher price of £7,676.45 per 10mg vial.

This means the two drugs would both cost approximately £300,000 for a year's treatment at their list price, but Akcea has clearly been quicker to offer an increased discount and secure a ‘yes’ from NICE.

Analysts say Akcea will be wise to offer greater discounts in order to gain market share, especially as its drug carries a safety warning for thrombocytopenia and renal toxicity which its rival does not.

Both Onpattro and Tegsedi also face competition from Pfizer’s Tafadamis, which has been available in Europe for a number of years as a polyneuropathy treatment, but is now set to gain approval in hATTR in the US and Europe.

NICE’s ruling adds to the good news for Akcea, which in March gained a CHMP recommendation for Waylivra (volanesorsen), which would be its second drug to reach market.

"We are delighted with this news that patients can access inotersen in England,” commented Dr. Richard A. Jones, SVP Head of Europe for Akcea Therapeutics. “hATTR amyloidosis is a debilitating disease that, to date, has had limited treatment options in the UK. We hope that other health technology assessment and reimbursement agencies across Europe will take NICE’s lead in making inotersen available as a treatment option for patients with this disease."

Article by
Andrew McConaghie

16th April 2019

From: Healthcare



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